Alex's Lemonade Stand Foundation for Childhood Cancer

Pediatric high-grade gliomas are among the deadliest childhood cancers. Survival has not improved in decades because these tumors grow quickly, resist standard treatments, and often return even after surgery, radiation, or chemotherapy. A small subset of these tumors carries an H3K27M mutation and shows high levels of a molecule called GD2 on the tumor surface. Early clinical trials using GD2-targeting CAR T cells, immune cells engineered to recognize GD2, have shown encouraging responses in these children. However, most high-grade gliomas do not carry this mutation.

Ewing sarcoma is a rare but deadly cancer that affects children and adolescents. It has a very poor prognosis and currently lacks effective treatment options. In recent years, a new type of therapy named “CAR T cells” has shown great results in several types of pediatric cancer.

Pediatric acute myeloid leukemia (AML) is a type of blood cancer affecting children and adolescents. Despite advances in therapy, survival has improved only modestly over the past decades, and relapse or treatment-resistant disease remains deadly. In AML, genetic changes can hijack the cell’s normal control systems, making key “switchboard operators” - proteins called transcription factors that coordinate multiple cellular programs - drive cells toward cancerous growth. Targeting these switchboard operators directly has proven extremely difficult.

Children with relapsed neuroblastoma have poor outcomes. The combination of GD2 antibodies and chemotherapy (called "chemoimmunotherapy") has shown a high response rate in this population. Chemoimmunotherapy often requires patients be admitted to the hospital, reducing quality of life for these children. This ongoing clinical trial is evaluating different infusion durations of a GD2 antibody called naxitamab together with chemotherapy to determine if this approach might be appropriate to deliver in an outpatient setting.